Category Archives: Gene therapy

Potent growth-inhibitory effect of a dual cancer-specific oncolytic adenovirus expressing apoptin on prostate carcinoma.
Int J Oncol. 2013 Mar;42(3):1052-60
Authors: Zhang M, Wang J, Li C, Hu N, Wang K, Ji H, He D, Quan C, Li X, Jin N, Li Y
Abstract
Apoptin is a chicken anemia virus-derived, p53-independent, bcl-2-insensitive apoptotic protein with the ability to specifically induce apoptosis in various human tumor cells, but not in normal cells. To explore the use of apoptin in tumor gene therapy, we assessed a recombinant adenovirus expressing the apoptin protein (Ad-hTERTp-E1a-Apoptin) in order to determine its lethal and growth-inhibitory effects on PC-3 and RM-1 cells in vitro and its antitumor effect on solid tumors in vivo....

Source:
http://www.medworm.com/index.php?rid=7005905&cid=c_449_6_f&fid=36721&url=http%3A%2F%2Fwww.ncbi.nlm.nih.gov%2FPubMed%2F23338489%3Fdopt%3DAbstract

Authors: Kurosaki T, Uematsu M, Shimoda K, Suzuma K, Nakai M, Nakamura T, Kitahara T, Kitaoka T, Sasaki H
Abstract
In this experiment, we developed anionic ternary complexes for effective and safe ocular gene delivery. Ternary complexes were constructed by coating plasmid DNA (pDNA)/polyethylenimine (PEI) complex with anionic polymers such as ?-polyglutamic acid (?-PGA) and chondroitin sulfate (CS). The cationic pDNA/PEI complex showed high gene expression on the human retinal pigment epithelial cell line, ARPE-19 cells. The pDNA/PEI complexes, however, also showed high cytotoxicity on the cells and aggregated strongly in the vitreous body. On the other hand, the anionic ternary complexes showed high gene expression on ARPE-19 cells without such cytotoxicity and aggregation. Afte...

Source:
http://www.medworm.com/index.php?rid=7013349&cid=c_449_13_f&fid=32516&url=http%3A%2F%2Fwww.ncbi.nlm.nih.gov%2FPubMed%2F23302641%3Fdopt%3DAbstract

Authors: Kurosaki T, Uematsu M, Shimoda K, Suzuma K, Nakai M, Nakamura T, Kitahara T, Kitaoka T, Sasaki H
Abstract
In this experiment, we developed anionic ternary complexes for effective and safe ocular gene delivery. Ternary complexes were constructed by coating plasmid DNA (pDNA)/polyethylenimine (PEI) complex with anionic polymers such as ?-polyglutamic acid (?-PGA) and chondroitin sulfate (CS). The cationic pDNA/PEI complex showed high gene expression on the human retinal pigment epithelial cell line, ARPE-19 cells. The pDNA/PEI complexes, however, also showed high cytotoxicity on the cells and aggregated strongly in the vitreous body. On the other hand, the anionic ternary complexes showed high gene expression on ARPE-19 cells without such cytotoxicity and aggregation. Afte...

Source:
http://www.medworm.com/index.php?rid=7013349&cid=c_449_13_f&fid=32516&url=http%3A%2F%2Fwww.ncbi.nlm.nih.gov%2FPubMed%2F23302641%3Fdopt%3DAbstract

Potent growth-inhibitory effect of a dual cancer-specific oncolytic adenovirus expressing apoptin on prostate carcinoma.
Int J Oncol. 2013 Mar;42(3):1052-60
Authors: Zhang M, Wang J, Li C, Hu N, Wang K, Ji H, He D, Quan C, Li X, Jin N, Li Y
Abstract
Apoptin is a chicken anemia virus-derived, p53-independent, bcl-2-insensitive apoptotic protein with the ability to specifically induce apoptosis in various human tumor cells, but not in normal cells. To explore the use of apoptin in tumor gene therapy, we assessed a recombinant adenovirus expressing the apoptin protein (Ad-hTERTp-E1a-Apoptin) in order to determine its lethal and growth-inhibitory effects on PC-3 and RM-1 cells in vitro and its antitumor effect on solid tumors in vivo....

Source:
http://www.medworm.com/index.php?rid=7005905&cid=c_449_6_f&fid=36721&url=http%3A%2F%2Fwww.ncbi.nlm.nih.gov%2FPubMed%2F23338489%3Fdopt%3DAbstract

Research on human beings has expanded greatly due to progress and the evolution of society as well as customs. Not only the unceasing development of research on human beings, but also interference in the beginning and end of life with homologous and heterogonous human reproduction, surrogate motherhood, cloning, gene therapies, eugenics,euthanasia, dysthanasia, orthothanasia, assisted suicide, genetic engineering, reassignment surgery in cases of transsexuality, the use of recombinant DNA technology and embryonic stem cells, transplantation of human organs and tissues, biotechnology and many other scientific advances. Scientific progress goes faster than the real needs of human beings, who are the final recipient of the entire evolutionary progress. Hence, there is the need to scrutinize w...

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http://www.medworm.com/index.php?rid=6986259&cid=c_449_19_f&fid=37449&url=http%3A%2F%2Fwww.scielo.br%2Fscielo.php%3Fscript%3Dsci_arttext%26pid%3DS1516-84842012000600016%26lng%3Den%26nrm%3Diso%26tlng%3Den

Research on human beings has expanded greatly due to progress and the evolution of society as well as customs. Not only the unceasing development of research on human beings, but also interference in the beginning and end of life with homologous and heterogonous human reproduction, surrogate motherhood, cloning, gene therapies, eugenics,euthanasia, dysthanasia, orthothanasia, assisted suicide, genetic engineering, reassignment surgery in cases of transsexuality, the use of recombinant DNA technology and embryonic stem cells, transplantation of human organs and tissues, biotechnology and many other scientific advances. Scientific progress goes faster than the real needs of human beings, who are the final recipient of the entire evolutionary progress. Hence, there is the need to scrutinize w...

Source:
http://www.medworm.com/index.php?rid=6986259&cid=c_449_19_f&fid=37449&url=http%3A%2F%2Fwww.scielo.br%2Fscielo.php%3Fscript%3Dsci_arttext%26pid%3DS1516-84842012000600016%26lng%3Den%26nrm%3Diso%26tlng%3Den

Public release date: 24-Jan-2013 [ | E-mail | Share ]

Contact: Gregory Harris gregory.harris@albertahealthservices.ca 403-619-3108 Alberta Health Services

CALGARY Researchers in Calgary have launched the first gene therapy clinical trial in the world for Fabry disease, a rare inherited enzyme deficiency that can shorten the lifespan of people who have it by as much as 40 years.

Researchers will first remove a quantity of stem cells from a Fabry patient's blood. Then a working copy of a new gene will be inserted into the stem cells using a specially engineered virus. During the final phase of the trial, researchers hope to transplant these stem cells back into the donor patient and the new, working copy of the gene will make the missing enzyme.

The clinical trial has been prompted by promising gene therapy results in mice performed in the laboratory of Dr. Jeffrey Medin at the University Health Network in Toronto. Dr. Medin is the principal investigator of the pan-Canada team grant that is supporting this trial.

"We hope this will one day become a form of treatment that effectively cures Fabry disease," says Dr. Aneal Khan, a medical geneticist based at Alberta Children's Hospital, who is leading the Calgary segment of the national project.

"It could also help establish a platform on which we can create gene therapies for other illnesses and establish Calgary as a national leader in this experimental field of interventional genetics."

Although several gene therapies have been used in Canada for cancer, this study will be the first in the country to test a gene therapy for an inherited metabolic disorder.

People with Fabry disease have a change in a gene called GLA and can't make enough enzyme to break down a fatty substance called Gb3. The build-up of Gb3 can lead to problems in the kidneys, heart and brain. About 400 Canadians, including 25 Calgarians, have Fabry disease.

Although the project is headquartered in Toronto, physicians and scientists in Calgary will play a major role in the clinical trial. In particular, the lab at Foothills Medical Centre in Calgary has specialized expertise in the stem cell filtering process that will be used for the clinical trial.

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Canada launches first gene therapy trial for Fabry disease

NEWARK, Calif., Jan. 23, 2013 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) announced today that Ann Tsukamoto, Ph.D., Executive Vice President, Research and Development, will make a presentation on the Company's clinical development programs at the Phacilitate Cell & Gene Therapy Forum to be held January 28-30, in Washington, DC. Dr. Tsukamoto is scheduled to speak at 12:25 p.m. ET on Wednesday, January 30, as part of the session on "Clinical development updates from leading cell and gene therapy product candidates in the clinic for CNS indications."

The Phacilitate Cell & Gene Therapy Forum is a preeminent industry-led meeting designed to help advance regulatory, manufacturing, R&D and commercial strategies and drive cell and gene therapy products forward. The Forum enables executives from global cell therapy, gene therapy and tissue engineering companies, representatives of big pharma and big biotech, regulators and regulatory experts, and public and private investors to meet and share information on the leading edge of the regenerative medicine sector.

About StemCells, Inc.

StemCells, Inc. is engaged in the research, development, and commercialization of cell-based therapeutics and tools for use in stem cell-based research and drug discovery. The Company's lead therapeutic product candidate, HuCNS-SC(R) cells (purified human neural stem cells), is currently in development as a potential treatment for a broad range of central nervous system disorders. In a Phase I clinical trial in Pelizaeus-Merzbacher disease (PMD), a fatal myelination disorder in children, the Company has shown preliminary evidence of progressive and durable donor-derived myelination in all four patients transplanted with HuCNS-SC cells. The Company is conducting a Phase I/II clinical trial in chronic spinal cord injury in Switzerland and recently reported positive interim data for the first patient cohort. The Company is also conducting a Phase I/II clinical trial in dry age-related macular degeneration (AMD), and is pursuing preclinical studies in Alzheimer's disease. StemCells also markets stem cell research products, including media and reagents, under the SC Proven(R) brand. Further information about StemCells is available at http://www.stemcellsinc.com.

The StemCells, Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=7014

Apart from statements of historical fact, the text of this press release constitutes forward-looking statements within the meaning of the U.S. securities laws, and is subject to the safe harbors created therein. These statements include, but are not limited to, statements regarding the clinical development of its HuCNS-SC cells; the Company's ability to commercialize drug discovery and drug development tools; and the future business operations of the Company. These forward-looking statements speak only as of the date of this news release. The Company does not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Such statements reflect management's current views and are based on certain assumptions that may or may not ultimately prove valid. The Company's actual results may vary materially from those contemplated in such forward-looking statements due to risks and uncertainties to which the Company is subject, including those described under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the year ended December 31, 2011 and in its subsequent reports on Forms 10-Q and 8-K.

More here:
StemCells, Inc. to Present at Phacilitate Cell & Gene Therapy Forum

NEWARK, Calif., Jan. 23, 2013 (GLOBE NEWSWIRE) -- StemCells, Inc. (STEM) announced today that Ann Tsukamoto, Ph.D., Executive Vice President, Research and Development, will make a presentation on the Company's clinical development programs at the Phacilitate Cell & Gene Therapy Forum to be held January 28-30, in Washington, DC. Dr. Tsukamoto is scheduled to speak at 12:25 p.m. ET on Wednesday, January 30, as part of the session on "Clinical development updates from leading cell and gene therapy product candidates in the clinic for CNS indications."

The Phacilitate Cell & Gene Therapy Forum is a preeminent industry-led meeting designed to help advance regulatory, manufacturing, R&D and commercial strategies and drive cell and gene therapy products forward. The Forum enables executives from global cell therapy, gene therapy and tissue engineering companies, representatives of big pharma and big biotech, regulators and regulatory experts, and public and private investors to meet and share information on the leading edge of the regenerative medicine sector.

About StemCells, Inc.

StemCells, Inc. is engaged in the research, development, and commercialization of cell-based therapeutics and tools for use in stem cell-based research and drug discovery. The Company's lead therapeutic product candidate, HuCNS-SC(R) cells (purified human neural stem cells), is currently in development as a potential treatment for a broad range of central nervous system disorders. In a Phase I clinical trial in Pelizaeus-Merzbacher disease (PMD), a fatal myelination disorder in children, the Company has shown preliminary evidence of progressive and durable donor-derived myelination in all four patients transplanted with HuCNS-SC cells. The Company is conducting a Phase I/II clinical trial in chronic spinal cord injury in Switzerland and recently reported positive interim data for the first patient cohort. The Company is also conducting a Phase I/II clinical trial in dry age-related macular degeneration (AMD), and is pursuing preclinical studies in Alzheimer's disease. StemCells also markets stem cell research products, including media and reagents, under the SC Proven(R) brand. Further information about StemCells is available at http://www.stemcellsinc.com.

The StemCells, Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=7014

Apart from statements of historical fact, the text of this press release constitutes forward-looking statements within the meaning of the U.S. securities laws, and is subject to the safe harbors created therein. These statements include, but are not limited to, statements regarding the clinical development of its HuCNS-SC cells; the Company's ability to commercialize drug discovery and drug development tools; and the future business operations of the Company. These forward-looking statements speak only as of the date of this news release. The Company does not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Such statements reflect management's current views and are based on certain assumptions that may or may not ultimately prove valid. The Company's actual results may vary materially from those contemplated in such forward-looking statements due to risks and uncertainties to which the Company is subject, including those described under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the year ended December 31, 2011 and in its subsequent reports on Forms 10-Q and 8-K.

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StemCells, Inc. to Present at Phacilitate Cell & Gene Therapy Forum

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